Why This Bay Area Pharma Could Win the Hemophilia Race With 'One And Done' Drug

Published: Jul 13, 2017

Why This Bay Area Pharma Could Win the Hemophilia Race With 'One And Done' Drug July 12, 2017
By Mark Terry, BioSpace.com Breaking News Staff

San Rafael, Calif.-based BioMarin Pharmaceutical stock jumped yesterday after it released positive updated data of its Phase I/II trial of BMN 270 for hemophilia A.

The company presented data at the International Society on Thrombosis and Haemostasis (ISTH) 2017 Congress in Berlin, Germany yesterday. In the trial, 15 patients with severe hemophilia A received a single dose of BMN 270. Seven received a dose of 6e13 vg/kg; the other six were given a lower dose of 4e13 vg/kg. Two more patients were given lower doses as part of a dose escalation study, but no therapeutic efficacy was observed.

The seven patients receiving the high dose hit an average 129 percent Factor VIII activity level by week 24. By week 52, they had an average Factor VIII activity level of 104 percent, which is considered normal range. The six patients on the lower dose had an average 33 percent Factor VIII activity level by week 24, which is classified as mild range for hemophilia.

The company plans to advance the higher dose into a Phase III trial in the fourth quarter of this year.

Joseph Schwartz, an analyst with Leerink Partners, believes investors may be concerned that the dose is higher than needed. “The rationale,” he wrote in a note to investors, “we believe, is to deliver a true ‘one and done’ curative therapy with a high likelihood of achieving normal Factor VIII levels with long-lasting efficacy that can garner favorable decisions from the reimbursement discussions.”

Half of the patients on the low dose also received a tapering dose of corticosteroids to deal with an increase of the enzyme alanine aminotransferase in the blood.

Schwartz wrote, “Based on multiple hemophilia studies, elevations in alanine aminotransferase now appear to be a common theme. Although some investors may find corticosteroid intervention to be a lingering concern, we view its transient nature and lack of repercussion on Factor VIII levels as encouraging.”

The European Medicines Agency (EMA) had granted BioMarin access to its Priority Medicines (PRIME) regulatory initiative for BMN 270.

The company has designed and built its first gene therapy manufacturing plant in Novato, Calif. “BioMarin has built a manufacturing facility that can produce BMN 270 at the scale and quantity to support clinical development and projected commercial demand, if approved,” said Robert Baffi, executive vice president Technical Operations for BioMarin, in a statement. “We drew upon our expertise in manufacturing complex biologics to efficiently build one of the largest gene therapy manufacturing facilities in the world, which will allow us to control scheduling, quality and costs to facilitate rapid product development.”

“The data continue to build the clinical case for the potentially groundbreaking impact of BMN 270 gene therapy for treating patients with hemophilia A,” said John Pasi, the primary investigator for the Phase I/II clinical trials, in a statement.

Pasi, at Barts and the London School of Medicine and Dentistry and Haemophilia Clinical Director at Barts Health NHS Trust, added, “In addition to the clinical data showing meaningful improvement in bleeds and Factor VIII levels up to 52 weeks, the quality of life data from the patients at the highest dose demonstrate that the potential clinical benefit could also represent a tangible improvement in a patient’s quality of life.”

BioMarin are currently trading for $90.78.

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